Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Sep 6, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called metformin on patients with Myotonic Dystrophy Type 1 (also known as Steinert's Disease). The researchers believe that metformin could help improve muscle function, especially for tasks like walking and gripping things, which are challenging for people with this condition. They will compare metformin to a placebo, which is a harmless pill that looks like the real medication but doesn’t contain any active ingredients. The study will also look at how safe metformin is for participants and how it affects other aspects of health, such as breathing and quality of life.

To participate in this trial, individuals need to be between 18 and 70 years old and have a confirmed diagnosis of Myotonic Dystrophy Type 1. They should still be able to walk with some assistance, and women must use effective birth control during the study. Unfortunately, some people may not be eligible, such as those who are pregnant, have certain heart or lung problems, or are allergic to metformin. If chosen for the trial, participants can expect to undergo regular assessments to monitor their muscle strength and overall health throughout the study. This research aims to find new ways to help those living with this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • DM1 disease confirmed by genetic analysis
• • Men and women between 18 and 70 years of age.
• • Preserved walking abilities (stick assistance possible)
• • MIRS score 3 or 4
• • Women of childbearing potential under efficient contraception during treatment
• • Patient able to consent
• • All patients who have completed and signed the specific information and informed consent form
• • Affiliation to a social security system
• Exclusion Criteria:
• • Pregnant or breast-feeding women
• • Men with an intention to conceive a child during the time of the study
• • Contraindications to Metformin (hypersensitivity to metformin or to one of the excipients)
• * Respiratory:
• • Patient requiring tracheotomy or
• • Patient requiring non-invasive-ventilation: - more than 12 hours per day; - insufficiently ventilated
• • Creatinine clearance inferior to 50 ml/min
• * Cardiac:
• • Left ventricular ejection fraction below 35%
• • Conduction system disease on the electrocardiogram with PR interval \>200 ms or QRS duration \>110 ms without a pacemaker or an implantable defibrillator or cardiac electrophysiological study performed over the past 5 years
• • Third-degree or Second degree type II atrioventricular block without a pacemaker or an implantable defibrillator
• • Sustained ventricular tachycardia
• • Acute disease that may lead to tissue hypoxia